Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy
about
Murine muscle cell models for Pompe disease and their use in studying therapeutic approachesTargeting adeno-associated virus and adenoviral gene therapy for hepatocellular carcinomaNew insights into therapeutic options for Pompe diseaseImmunomodulatory gene therapy in lysosomal storage disordersThe human acid alpha-glucosidase gene is a novel target of the Notch-1/Hes-1 signaling pathway.Gene therapy progress and prospects: gene therapy of lysosomal storage disorders.β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease.Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.Linker molecules between laminins and dystroglycan ameliorate laminin-alpha2-deficient muscular dystrophy at all disease stagesImpaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expressionAntibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.Neural deficits contribute to respiratory insufficiency in Pompe disease.Therapeutic approaches in glycogen storage disease type II/Pompe Disease.Imino sugar glucosidase inhibitors as broadly active anti-filovirus agentsThe respiratory neuromuscular system in Pompe disease.Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice.Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery.Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors.Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.
P2860
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P2860
Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy
description
2001 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած
@hyw
2001 թվականի սեպտեմբերին հրատարակված գիտական հոդված
@hy
artículu científicu espublizáu en 2001
@ast
im September 2001 veröffentlichter wissenschaftlicher Artikel
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scientific journal article
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vedecký článok (publikovaný 2001/09/15)
@sk
vědecký článek publikovaný v roce 2001
@cs
wetenschappelijk artikel (gepubliceerd op 2001/09/15)
@nl
наукова стаття, опублікована у вересні 2001
@uk
مقالة علمية (نشرت في 15-9-2001)
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name
Conditional tissue-specific ex ...... mice: implications for therapy
@ast
Conditional tissue-specific ex ...... mice: implications for therapy
@en
Conditional tissue-specific ex ...... mice: implications for therapy
@nl
type
label
Conditional tissue-specific ex ...... mice: implications for therapy
@ast
Conditional tissue-specific ex ...... mice: implications for therapy
@en
Conditional tissue-specific ex ...... mice: implications for therapy
@nl
prefLabel
Conditional tissue-specific ex ...... mice: implications for therapy
@ast
Conditional tissue-specific ex ...... mice: implications for therapy
@en
Conditional tissue-specific ex ...... mice: implications for therapy
@nl
P2093
P356
P1476
Conditional tissue-specific ex ...... mice: implications for therapy
@en
P2093
J J Hopwood
K Nagaraju
K Umapathysivam
P304
P356
10.1093/HMG/10.19.2039
P577
2001-09-01T00:00:00Z